Good news for the hundreds of thousands of children worldwide who suffer from sickle-cell anemia, the often painful genetic disease that largely affects people of African descent. Here’s what Howard University and the National Institutes of Health announced back in May- but we wanted to alert readers to important information you may have missed:
“Clinical trials conducted at Howard University College of Medicine and 12 other medical institutions across the nation have discovered a treatment that dramatically reduces pain, inflammation and hospitalization time and eases other symptoms in young children suffering from sickle cell anemia.”
This is significant news because one of the chief symptoms of sickle cell anemia is persistent pain in muscles and joints that can often render victims immobile. In addition, the disease can lead to organ failure in serious cases.
The four-year study found that using the drug hydroxycarbamide (also called hydroxyurea) in children 8 to 19 months with sickle cell anemia was safe and significantly reduced episodes of pain and other physiological problems. The drug has been used in adults for several decades for a variety of cancer treatments and is approved for use in adults with sickle cell anemia.
In an interview, Jonathan Goldsmith, a deputy chief at the National Heart, Lung and Blood Institute at the National Institutes of Health, who worked on the study, said that the families who participated in the study were “heroes” for helping advance researchers’ knowledge in the disease, which affects between 70,000 and 100,000 people in the United States.
“This is the best treatment out there,” said Goldsmith, who added that researchers will follow the patients tested for the research to see what kind of long term impacts the treatment has on young children.
Sickle cell anemia involves red blood cells that develop abnormal - or sickled- shapes that are unable to pass through capillaries and slow down the flow of blood through the body. These sickled cells also break down faster than normal blood cells, leading to anemia. Complications also include strokes and premature death.
The findings were published in May in The Lancet, the world’s leading medical journal and suggest that use of hydroxycarbamide “should now be the standard of care for all children with sickle cell disease.” The study was funded by The US National Heart, Lung and Blood Institute; and the National Institute of Child Health and Human Development.
The results caused quite a stir in the medical community when they were released and were heralded in my scientific circles as a major breakthrough in the treatment of sickle cell anemia for you children. More than 300,000 babies are born each year with the disease, mainly in low and middle income countries.
“These findings are extremely encouraging,” David Weatherall, a British physician and researcher in professor of molecular genetics pathology and clinical medicine who started the Weatherall Institute of Molecular Medicine at Oxford, wrote in The Lancet. “Hydroxycarbamide is inexpensive and could certainly be made available in low-income countries in which sickle-cell anaemia is so common.”
Howard officials also said that “children who received the hydroxycarbamide therapy were half as likely to experience pain, five times less likely to have painful inflammation of the hands or feet and three times less likely to experience acute chest syndrome than the children who did not.”
Here’s how Dr. Sohail Rana, professor of pediatrics in the Department of Pediatrics at Howard University College of Medicine and director of pediatric hematology at Howard University Hospital and one of the investigators on the study described the results:
“This is a significant breakthrough in the treatment of this disease, particularly for our very young patients,” Rana said. “To be able to dramatically diminish the pain that these young children suffer because of this disease is truly an important step forward. We hope all clinicians will now adopt this treatment for their patients.”